TOC
【FDA Approval Alert】medac’s treosulfan (Grafapex) Approved for Conditioning in AML/MDS allo-HSCT (January 21, 2025)
On January 21, 2025, the U.S. FDA approved treosulfan (Grafapex) in combination with fludarabine as part of a conditioning regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adult and pediatric patients (≥1 year old) with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Approval was based on MC-FludT.14/L Trial II (NCT00822393).
Disease Overview: Treosulfan in AML/MDS Conditioning
Conditioning regimens aim to eradicate malignant cells prior to allo-HSCT. Treosulfan offers a less toxic alternative to busulfan while maintaining efficacy.
Treatment Summary
- Drug Name: treosulfan (Grafapex)
- Company: medac GmbH
- Approval Date: January 21, 2025
- Indication: Conditioning prior to allo-HSCT in AML/MDS (adults and ≥1 year old pediatrics)
- Mechanism of Action: Alkylating agent
- Pivotal Trial: MC-FludT.14/L Trial II (NCT00822393)
- Key Efficacy:
- Overall Survival: HR 0.67 (95% CI: 0.51–0.90) vs busulfan arm
- AML subgroup: HR 0.73 (95% CI: 0.51–1.06); MDS subgroup: HR 0.64 (95% CI: 0.40–1.02)
Patient-Friendly Summary
Grafapex is used before stem cell transplantation and may offer reduced side effects compared to traditional regimens.
M&A Information
- Subsidiary: Not applicable (medac in-house development)
- Acquisition Date: Not applicable
- Development Stage at Acquisition: Not applicable
- Investor Information: Private Company (medac GmbH)
Source
This approval information is based on data from Drugs@FDA (U.S. FDA) and AACR releases.
